So much more than a “pair of brown shoes”: Triumphs of patient and other stakeholder engagement in patient-centered outcomes research

This piece illustrates the “real world” experiences of patients and other stakeholder partners in research to help inform and inspire future patient-centered outcomes research (PCOR) efforts. The Patient-Centered Outcomes Research Institute (PCORI) was created in 2010 to fund research that helps patients, clinicians, and other healthcare stakeholders make informed health decisions. The first 50 funded PCORI Pilot Projects engaged patients, caregivers, parents, patient advocates, clinicians, and other non-traditional research stakeholders to serve in advisory and leadership positions on their research teams, many for the first time. In interviews with seven patients and other stakeholders, several lessons learned emerged, including how to build confidence over the course of a research project; how to offer translation and interpretation insights reflective of practical experience; how to understand the benefits and limitations to stakeholder participation; and how to positively influence the research process and study outcomes. By the completion of their Pilot Projects, the stakeholder partners profiled here considered themselves “empowered” research contributors. The authors are hopeful these stories will encourage more patients and other stakeholders to contribute their time and experiential learnings to improve the process, and results of, PCOR

The effect of increasing physical activity and/or omega-3 supplementation on fatigue in inflammatory bowel disease

Objective: Fatigue is frequently reported by patients with inflammatory bowel disease (IBD), irrespective of disease activity; however, evidence regarding fatigue management is limited. This study tested the effect of individualised advice to increase physical activity and/or omega-3 fatty acids supplementation, on IBD-related fatigue. Methods: A pilot study in patients with inactive IBD, utilising a randomised controlled 2x2 factorial design (four groups) compared baseline and post-intervention fatigque scores. Study interventions: individualised exercise advice (15 minute consultation) and/or supplementation (omega-3 fatty acids, 2970mg/day) for 12 weeks. Control interventions: general health discussion and/or placebo supplement. All patients received follow-up support. Primary outcome was fatigue measured by Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) scale score; secondary outcomes included change in Inflammatory Bowel Disease-Fatigue (IBD-F) scale score. Results: From n=656 screened patients, n=74 who met the selection criteria were randomised, n=60 commenced, and n=52 completed the study. Fatigue as tThe primary outcome fatigque, measured with FACIT-F, showed slight worsening in the omega-3 supplementation group (95%CI:-8.6-(-0.7);p=0.02), and no change in the exercise advice group (p=0.38). Reduced fatigue, measured by IBD-F score, was identified in the exercise group (95%CI:-3.8-(-0.2);p=0.03). One treatment-related adverse event (musculoskeletal pain) was reported with exercise. Conclusions: Advice to increase physical activity and omega-3 supplementation, singly or in combination, were shown to be safe and generally well-tolerated. There was no evidence of exercise-related adverse effects on gut-related symptoms, and some evidence of improvement in fatigue. The slight worsening of fatigue with omega-3 supplementation is unexplained. Regular moderate to -vigorous exercise may be a self-management option in IBD-related fatigue

Patient-Centered Outcomes Measurement: Does It Require Information From Patients?

Purpose: Since collecting outcome measure data from patients can be expensive, time-consuming, and subject to memory and nonresponse bias, we sought to learn whether outcomes important to patients can be obtained from data in the electronic health record (EHR) or health insurance claims. Methods: We previously identified 21 outcomes rated important by patients who had advanced imaging tests for back or abdominal pain. Telephone surveys about experiencing those outcomes 1 year after their test from 321 people consenting to use of their medical record and claims data were compared with audits of the participants’ EHR progress notes over the time period between the imaging test and survey completion. We also compared survey data with algorithmically extracted data from claims files for outcomes for which data might be available from that source. Results: Of the 16 outcomes for which patients’ survey responses were considered to be the best information source, only 2 outcomes for back pain and 3 for abdominal pain had kappa scores above a very modest level of ≥ 0.2 for chart audit of EHR data and none for algorithmically obtained EHR/claims data. Of the other 5 outcomes for which claims data were considered to be the best information source, only 2 outcomes from patient surveys and 3 outcomes from chart audits had kappa scores ≥ 0.2. Conclusions: For the types of outcomes studied here, medical record or claims data do not provide an adequate source of information except for a few outcomes where patient reports may be less accurate

Protocol for the phase 2 EDELIFE trial investigating the efficacy and safety of intra-amniotic ER004 administration to male subjects with X-linked hypohidrotic ectodermal dysplasia

X-linked hypohidrotic ectodermal dysplasia (XLHED) is a rare genetic disorder characte-rised by abnormal development of the skin and its appendages, such as hair and sweat glands, the teeth, and mucous glands of the airways, resulting in serious, sometimes life-threatening complications like hyperthermia or recurrent respiratory infections. It is caused by pathogenic variants of the ectodysplasin A gene

Can incontinence be cured? A systematic review of cure rates

Background Incontinence constitutes a major health problem affecting millions of people worldwide. The present study aims to assess cure rates from treating urinary (UI) or fecal incontinence (FI) and the number of people who may remain dependent on containment strategies. Methods Medline, Embase, PsycINFO, Cochrane Central Register of Controlled Trials (CENTRAL), CINAHL, and PEDro were searched from January 2005 to June 2015. Supplementary searches included conference abstracts and trials registers (2013–2015). Included studies had patients ≥ 18 years with UI or FI, reported treatment cure or success rates, had ≥ 50 patients treated with any intervention recognized in international guideline algorithms, a follow-up ≥ 3 months, and were published from 2005 onwards. Title and abstract screening, full paper screening, data extraction and risk-of-bias assessment were performed independently by two reviewers. Disagreements were resolved through discussion or referral to a third reviewer where necessary. A narrative summary of included studies is presented. Results Most evidence was found for UI: Surgical interventions for stress UI showed a median cure rate of 82.3% (interquartile range (IQR), 72–89.5%); people with urgency UI were mostly treated using medications (median cure rate for antimuscarinics = 49%; IQR, 35.6–58%). Pelvic floor muscle training and bulking agents showed lower cure rates for UI. Sacral neuromodulation for FI had a median cure rate of 38.6% (IQR, 35.6–40.6%). Conclusions Many individuals were not cured and hence may continue to rely on containment. No studies were found assessing success of containment strategies. There was a lack of data in the disabled and in those with neurological diseases, in the elderly and those with cognitive impairment. Surgical interventions were effective for stress UI. Other interventions for UI and FI showed lower cure rates. Many individuals are likely to be reliant on containment strategies

The implementation of the serial trial intervention for pain and challenging behaviour in advanced dementia patients (STA OP!): a clustered randomized controlled trial

<p>Abstract</p> <p>Background</p> <p>Pain (physical discomfort) and challenging behaviour are highly prevalent in nursing home residents with dementia: at any given time 45-80% of nursing home residents are in pain and up to 80% have challenging behaviour. In the USA Christine Kovach developed the serial trial intervention (STI) and established that this protocol leads to less discomfort and fewer behavioural symptoms in moderate to severe dementia patients. The present study will provide insight into the effects of implementation of the Dutch version of the STI-protocol (STA OP!) in comparison with a control intervention, not only on behavioural symptoms, but also on pain, depression, and quality of life. This article outlines the study protocol.</p> <p>Methods/Design</p> <p>The study is a cluster randomized controlled trial in 168 older people (aged >65 years) with mild or moderate dementia living in nursing homes. The clusters, Dutch nursing homes, are randomly assigned to either the intervention condition (training and implementation of the STA OP!-protocol) or the control condition (general training focusing on challenging behaviour and pain, but without the step-wise approach). Measurements take place at baseline, after 3 months (end of the STA OP! training period) and after 6 months.</p> <p>Primary outcome measures are symptoms of challenging behaviour (measured with the Cohen-Mansfield Agitation Inventory (CMAI) and the Neuropsychiatric Inventory-Nursing Home version (NPI-NH)), and pain (measure with the Dutch version of the Pain Assessment Checklist for Seniors (PACSLAC-D) and the Minimum Data Set of the Resident Assessment Instrument (MDS-RAI) pain scale). Secondary outcome measures include symptoms of depression (Cornell and MDS-RAI depression scale), Quality of Live (Qualidem), changes in prescriptions of analgesics and psychotropic drugs, and the use of non-pharmacological comfort interventions (e.g. snoezelen, reminiscence therapy).</p> <p>Discussion</p> <p>The transfer from the American design to the Dutch design involved several changes due to the different organisation of healthcare systems. Specific strengths and limitations of the study are discussed.</p> <p>Trial registration</p> <p>Netherlands Trial Register (NTR): <a href="http://www.trialregister.nl/trialreg/admin/rctview.asp?TC=1967">NTR1967</a></p